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Results for “Gaucher's Disease”

172 trials

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Showing 20 of 172 results

Large-scale testing (Phase 3)Study completedNCT00478647
What this trial is testing

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

Who this might be right for
Gaucher Disease
Shire 40
Not applicableUnknownNCT02000310
What this trial is testing

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage Disorders
O & O Alpan LLC 80
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Not applicableStudy completedNCT00001289
What this trial is testing

Effects of Enzyme Replacement in Gaucher's Disease

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 70
Not applicableLooking for participantsNCT03190837
What this trial is testing

A Long-term Follow-up Study of Gaucher Disease

Who this might be right for
Gaucher Disease
Duke University 200
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Not applicableStudy completedNCT04845958
What this trial is testing

A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen

Who this might be right for
Gaucher Disease, SplenomegalyAcid SphingoMyelinase Deficiency
Sanofi 60
Not applicableUnknownNCT01716741
What this trial is testing

Identification of Undiagnosed Gaucher Disease

Who this might be right for
Gaucher Disease
Brigham and Women's Hospital 100
Large-scale testing (Phase 3)Study completedNCT00705939
What this trial is testing

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Who this might be right for
Gaucher Disease
Pfizer 45
Not applicableLooking for participantsNCT04388969
What this trial is testing

World Data on Ambroxol for Patients With GD and GBA Related PD

Who this might be right for
Gaucher DiseaseParkinson DiseaseGBA Gene Mutation
Shaare Zedek Medical Center 300
Not applicableLooking for participantsNCT05536388
What this trial is testing

Drug Discovery for Parkinson's With Mutations in the GBA Gene

Who this might be right for
Parkinson DiseaseGaucher DiseaseHealthy+1 more
New York Stem Cell Foundation Research Institute 60
Not applicableLooking for participantsNCT06162338
What this trial is testing

The Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I

Who this might be right for
Gaucher Disease Type I
He Huang 3
Not applicableStudy completedNCT05641103
What this trial is testing

PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"

Who this might be right for
Gaucher DiseaseAcid SphingoMyelinase DeficiencyASMD+2 more
Fundación Española de Hematología y Hemoterapía 122
Testing effectiveness (Phase 2)Study completedNCT00001416
What this trial is testing

Bone Response to Enzyme Replacement in Gaucher's Disease

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 100
Early research (Phase 1)Study completedNCT05324943
What this trial is testing

A Gene Therapy Study in Patients With Gaucher Disease Type 1

Who this might be right for
Gaucher Disease, Type 1
Spur Therapeutics 10
Testing effectiveness (Phase 2)Ended earlyNCT03746587
What this trial is testing

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Who this might be right for
Gaucher Disease, Type 1Gaucher Disease, Type 3
ZevraDenmark 39
Not applicableStudy completedNCT05702814
What this trial is testing

A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition

Who this might be right for
Gaucher Disease
Takeda 125
Testing effectiveness (Phase 2)Active Not RecruitingNCT02843035
What this trial is testing

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Who this might be right for
Gaucher Disease Type 1Gaucher Disease Type 3
Genzyme, a Sanofi Company 12
Testing effectiveness (Phase 2)Ended earlyNCT04145037
What this trial is testing

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
AVROBIO 8
Post-approval studies (Phase 4)UnknownNCT03721627
What this trial is testing

Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.

Who this might be right for
Gaucher DiseaseHCV
Mansoura University Children Hospital 10
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